WO2021011648A3 - Hydrogels fluides chargés d'acides nucléiques et compositions, systèmes et procédés associés - Google Patents

Hydrogels fluides chargés d'acides nucléiques et compositions, systèmes et procédés associés Download PDF

Info

Publication number
WO2021011648A3
WO2021011648A3 PCT/US2020/042131 US2020042131W WO2021011648A3 WO 2021011648 A3 WO2021011648 A3 WO 2021011648A3 US 2020042131 W US2020042131 W US 2020042131W WO 2021011648 A3 WO2021011648 A3 WO 2021011648A3
Authority
WO
WIPO (PCT)
Prior art keywords
compositions
systems
nucleic acid
methods related
acid loaded
Prior art date
Application number
PCT/US2020/042131
Other languages
English (en)
Other versions
WO2021011648A2 (fr
Inventor
Tatiana Segura
Evan KURT
Original Assignee
Duke University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Duke University filed Critical Duke University
Priority to US17/627,440 priority Critical patent/US20220409746A1/en
Priority to EP20841093.6A priority patent/EP3999137A4/fr
Publication of WO2021011648A2 publication Critical patent/WO2021011648A2/fr
Publication of WO2021011648A3 publication Critical patent/WO2021011648A3/fr

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/06Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005Wall or coating material
    • A61K9/5021Organic macromolecular compounds
    • A61K9/5036Polysaccharides, e.g. gums, alginate; Cyclodextrin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L27/00Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/50Materials characterised by their function or physical properties, e.g. injectable or lubricating compositions, shape-memory materials, surface modified materials
    • A61L27/54Biologically active materials, e.g. therapeutic substances
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/0062General methods for three-dimensional culture
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Cell Biology (AREA)
  • Oral & Maxillofacial Surgery (AREA)
  • Transplantation (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La présente invention concerne, en partie, des hydrogels fluides chargés d'acides nucléiques et des compositions, des systèmes et des procédés associés à ceux-ci, pour administrer efficacement des acides nucléiques à des cellules qui entrent en contact avec les hydrogels fluides.
PCT/US2020/042131 2019-07-15 2020-07-15 Hydrogels fluides chargés d'acides nucléiques et compositions, systèmes et procédés associés WO2021011648A2 (fr)

Priority Applications (2)

Application Number Priority Date Filing Date Title
US17/627,440 US20220409746A1 (en) 2019-07-15 2020-07-15 Nucleic acid loaded flowable hydrogels and compositions, systems and methods related thereto
EP20841093.6A EP3999137A4 (fr) 2019-07-15 2020-07-15 Hydrogels fluides chargés d'acides nucléiques et compositions, systèmes et procédés associés

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962874074P 2019-07-15 2019-07-15
US62/874,074 2019-07-15

Publications (2)

Publication Number Publication Date
WO2021011648A2 WO2021011648A2 (fr) 2021-01-21
WO2021011648A3 true WO2021011648A3 (fr) 2021-02-25

Family

ID=74211316

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2020/042131 WO2021011648A2 (fr) 2019-07-15 2020-07-15 Hydrogels fluides chargés d'acides nucléiques et compositions, systèmes et procédés associés

Country Status (3)

Country Link
US (1) US20220409746A1 (fr)
EP (1) EP3999137A4 (fr)
WO (1) WO2021011648A2 (fr)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113862332A (zh) * 2021-09-17 2021-12-31 浙江大学 琼脂糖在制备生物大分子冻干保护剂中的应用
CN114404646B (zh) * 2021-12-28 2022-10-11 安徽大学 CM-β-CD负载鞣酸聚丙烯酰胺型双网络抗菌水凝胶

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140227327A1 (en) * 2011-04-28 2014-08-14 President And Fellows Of Harvard College Injectable Cryogel Vaccine Devices and Methods of Use Thereof
KR101871241B1 (ko) * 2017-04-27 2018-06-28 한남대학교 산학협력단 성대 마비 치료용 고분자 주사 제제

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8883208B2 (en) * 2009-04-08 2014-11-11 Surmodics, Inc. Particles for delivery of nucleic acids and related devices and methods
CN107660220A (zh) * 2015-04-02 2018-02-02 密歇根大学董事会 超支化聚合物和多聚复合物及包含其的dna或rna递送系统

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140227327A1 (en) * 2011-04-28 2014-08-14 President And Fellows Of Harvard College Injectable Cryogel Vaccine Devices and Methods of Use Thereof
KR101871241B1 (ko) * 2017-04-27 2018-06-28 한남대학교 산학협력단 성대 마비 치료용 고분자 주사 제제

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JHA AMIT K., MALIK MANISHA S., FARACH-CARSON MARY C., DUNCAN RANDALL L., JIA XINQIAO: "Hierarchically structured, hyaluronic acid-based hydrogel matrices via the covalent integration of microgels into macroscopic networks", SOFT MATTER., vol. 6, no. 20, 15 June 2010 (2010-06-15), pages 5045 - 5055, XP055795335 *
SIDERIS ELIAS, GRIFFIN DONALD R., DING YICHEN, LI SHUORAN, WEAVER WESTBROOK M., DI CARLO DINO, HSIAI TZUNG, SEGURA TATIANA: "Particle Hydrogels Based on Hyaluronic Acid Building Blocks", ACS BIOMATER. SCI. ENG., vol. 2, 2016, pages 2034 - 2041, XP055795340 *

Also Published As

Publication number Publication date
US20220409746A1 (en) 2022-12-29
WO2021011648A2 (fr) 2021-01-21
EP3999137A2 (fr) 2022-05-25
EP3999137A4 (fr) 2023-08-09

Similar Documents

Publication Publication Date Title
WO2017184786A8 (fr) Complexes cpf1 à activité d'indel réduite
EP4361261A3 (fr) Nouvelles enzymes crispr orthologues cas13b et systèmes
AU2018251801A8 (en) Novel type VI CRISPR orthologs and systems
WO2017127807A8 (fr) Structure cristalline de crispr cpf1
EP3009511A3 (fr) Nouveaux systèmes et enzymes de crispr
WO2017027423A9 (fr) Compositions de crispr-cas9 manipulées et procédés d'utilisation
WO2020102503A3 (fr) Compositions de fusosome pour administration à des lymphocytes t
WO2017070633A3 (fr) Protéines cas9 évoluées pour l'édition génétique
WO2019006471A3 (fr) Nouveaux enzymes de ciblage d'arn crispr, systèmes et utilisations associés
WO2018213661A3 (fr) Agents de passivation pour cellules électrochimiques
SA522433260B1 (ar) Rsv f طافرات بروتين
WO2020102499A3 (fr) Compositions à base de fusosomes pour l'administration de snc
EP4257690A3 (fr) Polynucléotides adn/arn crispr hybrides et leurs procédés d'utilisation
WO2017020023A3 (fr) Acides nucléiques et procédés de détection d'anomalies chromosomiques
CA3010891A1 (fr) Brins d'acides nucleiques recombinogeniques in situ
WO2017066497A3 (fr) Ingénierie génomique avec systèmes crispr de type i dans des cellules eucaryotes
WO2016109840A3 (fr) Compositions et procédés pour une efficacité élevée de l'édition du génome in vivo
WO2016182959A8 (fr) Systèmes crispr/cas9 optimisés et procédés d'édition de gènes dans des cellules souches
WO2018094356A3 (fr) Compositions et méthodes de modification d'acides nucléiques cibles
WO2015112534A3 (fr) Compositions et procédés pour moduler et réorienter des réponses immunitaires
WO2016138534A3 (fr) Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide
WO2021011648A3 (fr) Hydrogels fluides chargés d'acides nucléiques et compositions, systèmes et procédés associés
WO2020236972A3 (fr) Systèmes de ciblage d'acides nucléiques à constituants multiples autres que de classe i
WO2017087916A3 (fr) Biocapteurs de glucose thermostables et leurs utilisations
WO2017103159A9 (fr) Polypeptides ayant une activité xylanase et polynucléotides codant pour ces polypeptides

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 20841093

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

ENP Entry into the national phase

Ref document number: 2020841093

Country of ref document: EP

Effective date: 20220215

121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 20841093

Country of ref document: EP

Kind code of ref document: A2